WHAT IS GSD1?
Glycogen Storage Disease Type 1 (GSD1) is a rare, genetic metabolic disorder that occurs when a specific enzyme is either missing or not functioning properly. This enzyme is responsible for maintaining the body's blood glucose (sugar) level. Glucose fuels every cell in our body, including brain activity. People affected with GSD1 cannot convert stored glycogen into glucose, and therefore need a constant external source of glucose in order to survive. Hypoglycemia in patients with GSD1 can be life threatening. GSD1 is the most common form Glycogen Storage Disease, affecting 1 in every 100,000 births. READ MORE
The Children's Fund for Glycogen Storage Disease Research is a public not-for-profit 501(c)(3) foundation that aims to make a difference in the lives of children and their families affected by GSD1.
As little as 40 years ago, a child born with GSD1 had very little chance of survival beyond one or two years. Today, thanks to increased awareness, medical advances and generous supporters, children are thriving and a cure is on the horizon.
Did you know…
News & Events
-
Pediatric Caregiver Interviews
Do you care for a child with GSD1a who is between the ages of 2-7? Ultragenyx wants to hear from you! If you would like to participate in a short interview, please send an email to gsdiapeds@discoveryworldwide.com expressing your interest. -
December 2023 Newsletter
Catch up on the latest GSD news and research from The Children's Fund. View our digital December 2023 Hopes and Dreams Newsletter below. -
Patient
SpotlightRunning a marathon is not an easy task for anyone. Running a marathon with GSD is something else entirely. With hard work, determination, and lots of smarties, this past November, Jake Gordon completed the NYC Marathon. Congrats Jake!
-
mRNA Trials
Underway!The first patient in the world received Moderna mRNA trial infusion for GSD1a at UConn Health. -
Investigational
mRNA TreatmentResearch is underway on an investigational mRNA treatment that could potentially correct the cause of GSD1a by teaching the body to break down glycogen. Click below to learn more about the Ba1ance Trial.
-
Gene Therapy
UpdateUltragenyx Therapeutics is now in Phase III clinical trials for GSD1a. The first participant recieved the infusion at Uconn Health in January 2022.
This template controls the elements:
FOOTER: Footer Title, Footer Descriptions
CUSTOM MENU: Images and columns into header main menu submenu items
* This message is only visible in administrative mode
The Children's Fund for
Glycogen Storage Disease Research
20 Sherwood Lane
Cheshire, CT 06410
" ... because every child deserves to be healthy."